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Dyslipidemia in Children Treated with a BRAF Inhibitor for Low-Grade Gliomas: A New Side Effect?
Marco Crocco1,2, Antonio Verrico1, Claudia Milanaccio1
1Neuroncology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Abstract:
BRAF inhibitors, in recent years, have played a central role in the disease control of unresectable BRAF-mutated pediatric low-grade gliomas (LGGs). The aim of the study was to investigate the acute and long-term effects of vemurafenib on the lipid metabolism in children treated for an LGG. In our cohort, children treated with vemurafenib (n = 6) exhibited alterations in lipid metabolism a few weeks after starting, as was demonstrated after 1 month (n = 4) by the high plasma levels of the total cholesterol (TC = 221.5 ± 42.1 mg/dL), triglycerides (TG = 107.8 ± 44.4 mg/dL), and low-density lipoprotein (LDL = 139.5 ± 51.5 mg/dL). Despite dietary recommendations, the dyslipidemia persisted over time. The mean lipid levels of the TC (222.3 ± 34.7 mg/dL), TG (134.8 ± 83.6 mg/dL), and LDL (139.8 ± 46.9 mg/dL) were confirmed abnormal at the last follow-up (45 ± 27 months, n = 6). Vemurafenib could be associated with an increased risk of dyslipidemia. An accurate screening strategy in new clinical trials, and a multidisciplinary team, are required for the optimal management of unexpected adverse events, including dyslipidemia.
Insights
Vemurafenib treatment in pediatric low-grade gliomas (LGGs) can cause significant lipid metabolism changes, including high cholesterol and triglycerides. These effects persist long-term, requiring careful monitoring and management in young patients.
Area of Science:
- Pediatric Oncology
- Pharmacology
- Metabolic Disorders
Background:
- BRAF inhibitors are crucial for treating unresectable BRAF-mutated pediatric low-grade gliomas (LGGs).
- Understanding the side effects of these treatments, particularly on metabolic health, is essential for patient management.
Purpose of the Study:
- To investigate the acute and long-term effects of vemurafenib on lipid metabolism in pediatric LGG patients.
- To assess the prevalence and persistence of dyslipidemia during vemurafenib therapy.
Main Methods:
- Observational study of pediatric patients with LGG treated with vemurafenib.
- Analysis of plasma lipid levels (total cholesterol, triglycerides, LDL) at baseline, 1 month, and last follow-up.
- Correlation of vemurafenib treatment duration with observed lipid alterations.
Main Results:
- Vemurafenib treatment led to significant alterations in lipid metabolism within weeks.
- Elevated total cholesterol (TC), triglycerides (TG), and low-density lipoprotein (LDL) were observed.
- These dyslipidemic changes persisted throughout the treatment duration, despite dietary interventions.
Conclusions:
- Vemurafenib is associated with an increased risk of dyslipidemia in pediatric LGG patients.
- Routine screening for lipid abnormalities and a multidisciplinary approach are necessary for managing vemurafenib-related adverse events.
- Further research into long-term metabolic consequences and management strategies is warranted.
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